Cystic fibrosis trial funding fears

Pioneering research into cystic fibrosis is being jeopardised because of funding problems caused by the economic downturn, a charity has said.

PUBLISHED: 05:57, Sun, Jun 5, 2011

Research into cystic fibrosis is being jeopardised because of funding problems, a charity has warned [PA]

The Cystic Fibrosis Trust needs to plug a £6 million funding gap so ground-breaking work into treating the genetic disease can continue.

UK researchers were hoping to begin a clinical trial later this summer using gene therapy to try to protect sufferers' lungs from potentially fatal damage. But this has been put back until Spring next year while fundraisers attempt to raise the deficit through donations.

Professor Eric Alton, who is heading the research, told the BBC the project could disintegrate without fresh backing. He said: "This project cannot be parked. If we do not get the funding patients will become more unwell. Some will die. A huge amount of hope and effort and science has been invested in this."

The UK CF Gene Therapy Consortium is hoping to begin a multi-dose clinical trial after spending the last decade looking at ways to threat the disease, which is caused by a faulty gene that controls the movement of salt and water through cells.

Scientists say it is a crucial stage of the research's development and follows successful tests on the safety and toxicity of a gene therapy product which they hope will threat the disease. If the results are positive they aim is to find a pharmaceutical partner to take the product for further tests.

"The chance of securing a multi-dose trial is a once-in-a-lifetime opportunity which we cannot miss," said the charity. "Without proceeding with this trial we would not know whether effective gene therapy for CF could become a reality."

Around £30 million has been spent on research so far.

The charity's chief executive, Matthew Reed, said around 9,000 people are affected by the disease.

"Cystic fibrosis undermines the quality and length of life," he said. "This is a really significant moment in the history of the disease. We are very close to a clinical trial to test for efficacy. A lot of people are doing all they can to make it happen."

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